AveXis Receives Orphan Drug Designation

AveXis has received Orphan Drug Designation from the FDA for their gene therapy program, called chariSMA. Orphan Drug Designation is granted by the FDA to encourage and expedite the development of drugs for conditions like SMA.

In the chariSMA program, researchers are using the AAV9 virus to “infect” a cell with new DNA from survival motor neuron gene 1 (SMN1). Researchers believe this treatment may be able to correct the SMN1 mutation that causes SMA.

Phase I Clinical Trial Enrollment

Currently, AveXis is recruiting participants for a Phase I clinical trial to test this approach using systemic delivery of the drug.

The trial is open to infants with SMA type I who are nine months of age or less, and who meet certain other criteria.

Cure SMA’s Support of Gene Therapy

In 2010, Cure SMA made an initial grant to Dr. Brian Kaspar to begin study of this approach, followed by a second grant in 2012 to allow the drug to treat older and bigger patients using a different delivery method directly into the central nervous system (CNS).

Using the data generated with our funding on the CNS delivery of the drug, Dr. Kaspar and his team were able to secure a $4 million grant from National Institute of Neurological Disorders and Stroke (NINDS) for additional development.

The technology for both systemic and CNS-delivered gene therapy was then licensed to AveXis in 2013 for clinical testing.

One of the most distinctive aspects of our research program is our seed funding strategy. We fund the early stages of a drug project, with the goal of attracting increased funding from government and pharmaceutical companies as the process moves forward.

This project represents a major victory for our seed funding approach. Through early investment in gene therapy, we have been able to attract support from the government through NINDS—and now, through the FDA—as well as from AveXis, a biotechnology partner.

The CNS-delivered drug is also advancing toward clinical trials, and we hope both routes of delivery will soon be tested in individuals with SMA.

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